Status:

RECRUITING

A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF

Lead Sponsor:

ReCode Therapeutics

Conditions:

Cystic Fibrosis

Eligibility:

All Genders

18-60 years

Phase:

PHASE2

Brief Summary

This is the first-in-human study with RCT2100 and is designed to provide safety and tolerability data for future clinical studies.

Detailed Description

This is a multi-part study to assess the safety, tolerability, and biodistribution of a single ascending dose of inhaled RCT2100 administered via nebulizer to healthy participants (Part 1), the safety...

Eligibility Criteria

Part 1 Major Inclusion Criteria:

  • Healthy, adult, male or female, 18-55 years of age, inclusive, at screening.
  • Body weight greater than or equal to 50 kg and body mass index (BMI) between 16-32 kg/m2, inclusive
  • The participant has a forced expiratory volume in one second (FEV1) of at least 80% predicted
  • The participant is considered by the investigator to be in good general health as determined by medical history, clinical laboratory test results, vital sign measurements, 12-lead ECG results, and physical examination findings at screening.
  • Understands the study procedures in the informed consent form (ICF), and is willing and able to comply with the protocol.

Part 1 Major Exclusion Criteria:

  • History or presence of clinically significant medical, surgical, clinical laboratory, or psychiatric condition or disease.
  • The participant has supine blood pressure (BP) >150 mm Hg (systolic) or >90 mm Hg (diastolic), following at least 5 minutes of supine rest.
  • The participant has abnormal clinical laboratory tests at screening, as assessed by the study-specific laboratory.
  • The participant is a smoker or has used nicotine or nicotine-containing products 6 weeks before the first dose of study drug. Former smokers with greater than 10 pack years of smoking history are excluded.

Part 2 Major Inclusion Criteria:

  • Confirmed diagnosis of CF
  • Forced expiratory volume in 1 second ≥50% and ≤100% of predicted mean value for age, sex, and height
  • a) Not eligible for CFTR modulators based on having mutations of CFTR gene on both alleles that are not responsive to CFTR modulator therapy OR
  • b) Eligible for CFTR modulators (based on local prescribing information) but not using CFTR modulators due to intolerance or contraindications

Part 2 Major Exclusion Criteria:

  • Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
  • An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for sinopulmonary disease within 4 weeks before the first dose of study drug
  • Lung infection with organisms associated with a more rapid decline in pulmonary status
  • Arterial oxygen saturation on room air less than 94% at screening
  • Treatment with a CFTR modulator (Kalydeco, Trikafta, Symdeko, Orkambi, or Alyftrek) within 12 weeks of Screening

Other protocol defined Inclusion/Exclusion criteria may apply.

Part 3 Major Inclusion Criteria:

  • Confirmed diagnosis of CF
  • Forced expiratory volume in 1 second ≥50% and ≤100% of predicted mean value for age, sex, and height
  • a) Not eligible for CFTR modulators based on having mutations of CFTR gene on both alleles that are not responsive to CFTR modulator therapy OR
  • b) Eligible for dual or triple CFTR modulators (based on local prescribing information) but not using CFTR modulators due to intolerance or contraindications

Part 3 Major Exclusion Criteria:

  • Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
  • An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for sinopulmonary disease within 4 weeks before the first dose of study drug
  • Lung infection with organisms associated with a more rapid decline in pulmonary status
  • Arterial oxygen saturation on room air less than 94% at screening
  • Treatment with a CFTR modulator (Kalydeco, Trikafta, Symdeko, Orkambi, or Alyftrek) within 12 weeks of Screening

Other protocol defined Inclusion/Exclusion criteria may apply.

Key Trial Info

Start Date :

February 1 2024

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

December 31 2026

Estimated Enrollment :

192 Patients enrolled

Trial Details

Trial ID

NCT06237335

Start Date

February 1 2024

End Date

December 31 2026

Last Update

January 16 2026

Active Locations (23)

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Page 1 of 6 (23 locations)

1

The University of Alabama at Birmingham

Birmingham, Alabama, United States, 35294

2

University of Arizona

Tucson, Arizona, United States, 85719

3

Stanford University

Palo Alto, California, United States, 94304

4

UCSD

San Diego, California, United States, 92037